title: Adopting nested case-control quota sampling designs for the evaluation of risk markers.
abstract: Two-phase study methods, in which more detailed or more expensive exposure information is only collected on a sample of individuals with events and a small proportion of other individuals, are expected to play a critical role in biomarker validation research. One major limitation of standard two-phase designs is that they are most conveniently employed with study cohorts in which information on longitudinal follow-up and other potential matching variables is electronically recorded. However for many practical situations, at the sampling stage such information may not be readily available for every potential candidates. Study eligibility needs to be verified by reviewing information from medical charts one by one. In this manuscript, we study in depth a novel study design commonly undertaken in practice that involves sampling until quotas of eligible cases and controls are identified. We propose semiparametric methods to calculate risk distributions and a wide variety of prediction indices when outcomes are censored failure times and data are collected under the quota sampling design. Consistency and asymptotic normality of our estimators are established using empirical process theory. Simulation results indicate that the proposed procedures perform well in finite samples. Application is made to the evaluation of a new risk model for predicting the onset of cardiovascular disease.
authors.affiliation: Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North, Seattle, WA, 98109, USA, yzheng@fhcrc.org.
authors.forename: Yingye
authors.initials: Y
authors.lastname: Zheng
authors.forename: Tianxi
authors.initials: T
authors.lastname: Cai
authors.forename: Margaret S
authors.initials: MS
authors.lastname: Pepe
mesh_terms: D015415:Biomarkers / Q000097:blood; D056808:Biostatistics; D002097:C-Reactive Protein / Q000032:analysis; D002318:Cardiovascular Diseases / Q000097:blood / Q000453:epidemiology / Q000209:etiology; D016022:Case-Control Studies; D006801:Humans; D008404:Massachusetts / Q000453:epidemiology; D015233:Models, Statistical; D011237:Predictive Value of Tests; D016016:Proportional Hazards Models; D012306:Risk; D012307:Risk Factors; D012494:Sampling Studies
chemical_list: D015415:Biomarkers; D002097:C-Reactive Protein